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PMID: 14576293 Published · ppublish English Journal Article Review

Therapeutic modulation of endogenous gene function by agents with designed DNA-sequence specificities.

Nucleic acids research ·Vol. 31 ·No. 21 ·2003-11-01 ·Pages 6064-78

Uil TG, Haisma HJ, Rots MG

Abstract

Designer molecules that can specifically target pre-determined DNA sequences provide a means to modulate endogenous gene function. Different classes of sequence-specific DNA-binding agents have been developed, including triplex-forming molecules, synthetic polyamides and designer zinc finger proteins. These different types of designer molecules with their different principles of engineered sequence specificity are reviewed in this paper. Furthermore, we explore and discuss the potential of these molecules as therapeutic modulators of endogenous gene function, focusing on modulation by stable gene modification and by regulation of gene transcription.

MeSH Terms
Animals DNA/chemistry,genetics,metabolism DNA-Binding Proteins/metabolism Gene Expression Regulation Humans Nylons/metabolism Substrate Specificity Transcription Factors/metabolism
Chemicals
DNA-Binding Proteins Nylons Transcription Factors triplex DNA DNA
Authors & Affiliations
3 authors, click to expand affiliations / ORCID
Uil Taco G
Department of Molecular Cell Biology, Leiden University Medical Center, Leiden, The Netherlands.
Haisma Hidde J
Rots Marianne G
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Article Info
Journal
Nucleic acids research
Abbr.
Nucleic Acids Res
ISSN
1362-4962
Published
2003-11-01
Pages
6064-78
Language
English
Region
England
NLM ID
0411011
PMCID
PMC275457
Subset
IM
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